Bronchiolitis ObliteransPhase 3BOSTON-3NCT04039347
Extension Trial on Efficacy / Safety of L-CsA + SoC in Treating BOS in Post Single or Double Lung Transplant (BOSTON-3)
A Phase 3 study testing Liposomal Cyclosporine A 5 mg and Liposomal Cyclosporine A 10 mg in people with Bronchiolitis Obliterans and Obliterative Bronchiolitis, run by Zambon SpA at 37 sites in 9 countries.
Trial summary
The objective of the trial is to assess the long-term safety and efficacy of L-CsA plus Standard of Care (SoC) in the treatment of BOS in single (SLT) and double lung transplant (DLT) recipients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bronchiolitis Obliterans, Obliterative Bronchiolitis, Bronchiolitis Obliterans Syndrome
- What is being tested?
- Liposomal Cyclosporine A 5 mg and Liposomal Cyclosporine A 10 mg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 164 participants
- Where
- 37 sitesin 9 countries
- Study dates
- 12 Mar 2020to 25 Feb 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who have completed all visits through the End of Treatment Visit in either BOSTON-1 or BOSTON-2, did not withdraw informed consent, and did not prematurely terminate study drug administration.
- Patients should be on a three-drug maintenance regimen of immunosuppressive agents including tacrolimus or another CNI, a second agent such as but not limited to MMF or azathioprine, and a systemic corticosteroid such as prednisone.
- Patients capable of understanding the purposes and risks of the clinical trial, who have given written informed consent and agree to comply with the clinical trial requirements/visit schedules, and who are capable of aerosol inhalation.
- Women of childbearing potential must have a negative serum pregnancy test within 7 days prior to Visit 1 and must agree to use one of the methods of contraception listed in Appendix II through their End of Study Visit.
Who cannot join
- Known hypersensitivity to L-CsA or to cyclosporine A.
- Patients who experienced an AE related to study drug that led to permanent study drug discontinuation in BOSTON-1 or BOSTON-2.
- Patients with new onset of malignancy while participating in BOSTON-1 or BOSTON-2, including post-transplant lymphoproliferative disorder, with the exception of treated, localized basal and squamous cell carcinomas.
- Pregnant women or women who are unwilling to use appropriate birth control to avoid pregnancy through their End of Study Visit.
- Women who are currently breastfeeding.
- Receipt of an investigational drug, other than L-CsA, as part of a clinical trial within 4 weeks prior to Visit 1. This is defined as any treatment that is implemented under an Investigational New Drug (IND) or compassionate use.
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- Patients who are currently participating in an interventional clinical trial, other than BOSTON-1 or BOSTON-2.
- Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary procedures.
- Any co-existing medical condition that in the Investigator's judgment will substantially increase the risk associated with the patient's participation in the clinical trial.
Trial locations and contacts
37 sites in 9 countries.
37 sites in 9 countries
United States20 sites
University of Maryland
Baltimore, Maryland, 21201
Johns Hopkins University Hospital
Baltimore, Maryland, 21287
Cleveland Clinic
Cleveland, Ohio, 44195
OSU Wexner Medical Center
Columbus, Ohio, 43210
Baylor University Medical Center
Dallas, Texas, 75246
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Duke University
Durham, North Carolina, 27710
University of Florida Dept of Pulmonary Medicine
Gainesville, Florida, 32610
Baylor College of Medicine
Houston, Texas, 77030
Houston Methodist Hospital
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
UK Albert B. Chandler Hospital
Lexington, Kentucky, 40508
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
Columbia University Medical Center
New York, New York, 10032
Temple University Hospital
Philadelphia, Pennsylvania, 19140
Banner - University Medical Center
Phoenix, Arizona, 85006
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15232
UCSF
San Francisco, California, 94143
Barnes-Jewish Hospital
St Louis, Missouri, 63110
UCSF Center for Advanced Lung Disease
Stanford, California, 94305
Spain5 sites
Complexo Hospitalario de A Coruna
A Coruña
Hospital Universitari Vall d'Hebron
Barcelona
Hospital Universitario Puerta de Hierro - Unidad de Trasplante Pulmonar
Madrid, 28222
Hospital Marques de Valdecilla
Santander
Unidad de Trasplante Pulmonar del Hospital La Fe
Valencia, 46026
France3 sites
Hopital Marie Lannelongue
Le Plessis-Robinson, 92350
CHU Hopital Nord
Marseille
Hopitaux Universitaires de Strasbourg
Strasbourg
Belgium2 sites
Hôpital Erasme
Brussels, 1070
Universitair Ziekenhuis Leuven
Leuven, 3000
United Kingdom2 sites
Royal Papworth Hospital NHS Foundation Trust
Cambridge, CB23 3RE
University of Manchester
Manchester
About this study
Official title: A Phase III, Extension Clinical Trial to Demonstrate Efficacy and Safety of Liposomal Cyclosprine A Via the PARI Investigational eFlow® Device and SoC in Treating Bronchiolitis Obliterans in Patients Post Single or Double Lung Transplant
The objective of the trial is to assess the long-term safety and efficacy of L-CsA plus Standard of Care (SoC) in the treatment of BOS in single (SLT) and double lung transplant (DLT) recipients.
Read the detailed description
This is a Phase III, multicenter, open-label, extension clinical trial of L-CsA for the treatment of BOS.
Enrollment will be limited to patients who have completed 48 weeks participation in either the BT-L-CsA-301-SLT (BOSTON-1) or BT-L-CsA-302-DLT (BOSTON-2) trial. All patients in this clinical trial will receive L-CsA in addition to SoC, regardless of the randomization arm in prior trials.
IMP will be administered by BID inhalation (morning/evening) using the L-CsA eFlow. Patients who did not receive L-CsA in BOSTON-1 or BOSTON-2 must remain in the clinic for at least 4 hours for observation after the first inhalation. At all subsequent visits, one dose administered via inhalation will be monitored by the clinical trial center personnel. In case patients receiving L-CsA undergo the last visit for BOSTON-1 or BOSTON-2 (Visit 9) on the same day as for Visit 1 for BOSTON-3, they will take the first dose for Boston 3 in the evening of this day. This first dose will not be supervised by the site staff. Nebulization time per inhalation dose is approximately 6-10 minutes for the 5 mg dose and 9-13 minutes for the 10 mg dose. Inhalations will be performed BID approximately 12 hours apart through a mouthpiece by slow and deep respiration using the L-CsA eFlow. A high efficiency particulate air filter is used to prevent environmental contamination during exhalation.
- Being tested
- Liposomal Cyclosporine A 5 mg, Liposomal Cyclosporine A 10 mg
- Keywords
- single and double transplant
- Medical subject headings
- Bronchiolitis Obliterans, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liposomal Cyclosporine A 5 mgDrug
Also called L-CsA
delivered via the PARI eFlow® device, which is a new technology of nebulizing liquid drugs with a perforated vibrating membrane resulting in an aerosol with a low ballistic momentum and a high percentage of droplets in a respirable size range of 3-5 μm
Liposomal Cyclosporine A 10 mgDrug
Also called L-CsA
delivered via the PARI eFlow® device, which is a new technology of nebulizing liquid drugs with a perforated vibrating membrane resulting in an aerosol with a low ballistic momentum and a high percentage of droplets in a respirable size range of 3-5 μm
| Study group | Role | What they receive |
|---|---|---|
| L-CsA 5 mg plus Standard of Care | Experimental | L-CsA 5 mg twice daily plus Standard of Care for up to 144 weeks for patients post Single Lung Transplant Receives: Drug: Liposomal Cyclosporine A 5 mg |
| L-CsA 10 mg plus Standard of Care | Experimental | L-CsA 10 mg twice daily plus Standard of Care for up to 144 weeks for patients post Double Lung Transplant Receives: Drug: Liposomal Cyclosporine A 10 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change in FEV1 from Baseline to Week 24
FEV1 is the Forced Expiratory Volume in One Second
Measured: Baseline to Week 24
Secondary outcomes (5)
Mean change in FEV1 from Baseline to Week 48
FEV1 is the Forced Expiratory Volume in One Second
Measured: Baseline to Week 48
Mean change in FEV1 from Baseline to End of Study
FEV1 is the Forced Expiratory Volume in One Second
Measured: Baseline to end of study, approximately 2 years
Mean change in FEV1/FVC from Baseline to Week 24
FEV1/FVC is the ratio between Forced Expiratory Volume in One Second and Forced Vital Capacity.
Measured: Baseline to Week 24
Mean change in FEV1/FVC from Baseline to Week 48
FEV1/FVC is the ratio between Forced Expiratory Volume in One Second and Forced Vital Capacity.
Measured: Baseline to Week 48
Time to Progression of BOS
The Progression of BOS is defined as the earliest of:
- Absolute decrease from baseline in FEV1 >/= 10% or >/= 200 mL and absolute decrease in FEV1/FVC of > 5%, OR
- Change in BOS severity (according to criteria in Verleden 2019), OR
- Re-transplantation, OR
- Death from respiratory failure
Measured: Baseline to End of Study, approximately 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2019
- Study start12 Mar 2020
- Primary completion25 Feb 2026
- Study completion25 Feb 2026
Why it stopped: Study was terminated for business reasons. The earlier study end is not related to any safety concerns or data issues identified at any site or within the overall study data collected to date.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bronchiolitis ObliteransStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 164 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zambon SpA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paola R Castellani, MD, Study director, Zambon SpA, Chief Medical Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04039347
- Sponsor's protocol ID
- BT - L-CsA - 303 - FU
- Other IDs
- 2019-002987-29, 2019
- Acronym
- BOSTON-3
- Status last verified
- Apr 2026
- First submitted
- 19 Jul 2019
- Last updated
- 7 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Extension Clinical Trial to Demonstrate Efficacy and Safety of Liposomal Cyclosprine A Via the PARI Investigational eFlow® Device and SoC in Treating Bronchiolitis Obliterans in Patients Post Single or Double Lung Transplant
- ClinicalTrials.gov ID
- NCT04039347
- Acronym / sponsor ID
- BOSTON-3 / BT - L-CsA - 303 - FU
- Keywords
- single and double transplant
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated for business reasons. The earlier study end is not related to any safety concerns or data issues identified at any site or within the overall study data collected to date.
- Enrolment
- 164 participants (actual)
- Lead sponsor
- Zambon SpAIndustry (pharma, biotech, devices)
- Study start
- 12 Mar 2020 (actual)
- Primary completion
- 25 Feb 2026 (actual)
- Study completion
- 25 Feb 2026 (actual)
- First posted
- 31 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of the trial is to assess the long-term safety and efficacy of L-CsA plus Standard of Care (SoC) in the treatment of BOS in single (SLT) and double lung transplant (DLT) recipients.
Detailed description
This is a Phase III, multicenter, open-label, extension clinical trial of L-CsA for the treatment of BOS.
Enrollment will be limited to patients who have completed 48 weeks participation in either the BT-L-CsA-301-SLT (BOSTON-1) or BT-L-CsA-302-DLT (BOSTON-2) trial. All patients in this clinical trial will receive L-CsA in addition to SoC, regardless of the randomization arm in prior trials.
IMP will be administered by BID inhalation (morning/evening) using the L-CsA eFlow. Patients who did not receive L-CsA in BOSTON-1 or BOSTON-2 must remain in the clinic for at least 4 hours for observation after the first inhalation. At all subsequent visits, one dose administered via inhalation will be monitored by the clinical trial center personnel. In case patients receiving L-CsA undergo the last visit for BOSTON-1 or BOSTON-2 (Visit 9) on the same day as for Visit 1 for BOSTON-3, they will take the first dose for Boston 3 in the evening of this day. This first dose will not be supervised by the site staff. Nebulization time per inhalation dose is approximately 6-10 minutes for the 5 mg dose and 9-13 minutes for the 10 mg dose. Inhalations will be performed BID approximately 12 hours apart through a mouthpiece by slow and deep respiration using the L-CsA eFlow. A high efficiency particulate air filter is used to prevent environmental contamination during exhalation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| L-CsA 5 mg plus Standard of Care | Experimental | Drug: Liposomal Cyclosporine A 5 mgL-CsA 5 mg twice daily plus Standard of Care for up to 144 weeks for patients post Single Lung Transplant |
| L-CsA 10 mg plus Standard of Care | Experimental | Drug: Liposomal Cyclosporine A 10 mgL-CsA 10 mg twice daily plus Standard of Care for up to 144 weeks for patients post Double Lung Transplant |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients who have completed all visits through the End of Treatment Visit in either BOSTON-1 or BOSTON-2, did not withdraw informed consent, and did not prematurely terminate study drug administration.
- Patients should be on a three-drug maintenance regimen of immunosuppressive agents including tacrolimus or another CNI, a second agent such as but not limited to MMF or azathioprine, and a systemic corticosteroid such as prednisone.
- Patients capable of understanding the purposes and risks of the clinical trial, who have given written informed consent and agree to comply with the clinical trial requirements/visit schedules, and who are capable of aerosol inhalation.
- Women of childbearing potential must have a negative serum pregnancy test within 7 days prior to Visit 1 and must agree to use one of the methods of contraception listed in Appendix II through their End of Study Visit.
Key exclusion criteria (9 listed)
- Known hypersensitivity to L-CsA or to cyclosporine A.
- Patients who experienced an AE related to study drug that led to permanent study drug discontinuation in BOSTON-1 or BOSTON-2.
- Patients with new onset of malignancy while participating in BOSTON-1 or BOSTON-2, including post-transplant lymphoproliferative disorder, with the exception of treated, localized basal and squamous cell carcinomas.
- Pregnant women or women who are unwilling to use appropriate birth control to avoid pregnancy through their End of Study Visit.
- Women who are currently breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Liposomal Cyclosporine A 5 mgAlso: L-CsAdelivered via the PARI eFlow® device, which is a new technology of nebulizing liquid drugs with a perforated vibrating membrane resulting in an aerosol with a low ballistic momentum and a high percentage of droplets in a respirable size range of 3-5 μm | Drug | L-CsA 5 mg plus Standard of Care |
| Liposomal Cyclosporine A 10 mgAlso: L-CsAdelivered via the PARI eFlow® device, which is a new technology of nebulizing liquid drugs with a perforated vibrating membrane resulting in an aerosol with a low ballistic momentum and a high percentage of droplets in a respirable size range of 3-5 μm | Drug | L-CsA 10 mg plus Standard of Care |
Primary outcome
Mean change in FEV1 from Baseline to Week 24
Time frame: Baseline to Week 24
FEV1 is the Forced Expiratory Volume in One Second
5 secondary outcomes
Mean change in FEV1 from Baseline to Week 48
Time frame: Baseline to Week 48
Mean change in FEV1 from Baseline to End of Study
Time frame: Baseline to end of study, approximately 2 years
Mean change in FEV1/FVC from Baseline to Week 24
Time frame: Baseline to Week 24
Mean change in FEV1/FVC from Baseline to Week 48
Time frame: Baseline to Week 48
Time to Progression of BOS
Time frame: Baseline to End of Study, approximately 2 years
Results and safety
The study was stopped early (reason given: Study was terminated for business reasons. The earlier study end is not related to any safety concerns or data issues identified at any site or within the overall study data collected to date.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paola R Castellani, MD study director
Zambon SpA, Chief Medical Officer
37 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Liposomal Cyclosporine A 5 mg and Liposomal Cyclosporine A 10 mg in people with Bronchiolitis Obliterans and Obliterative Bronchiolitis, run by Zambon SpA at 37 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Spain, France, Belgium, Germany and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study was terminated for business reasons. The earlier study end is not related to any safety concerns or data issues identified at any site or within the overall study data collected to date.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change in FEV1 from Baseline to Week 24 (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.